MEDIA ALERT: Two Studies Presented at ASH Demonstrate Quality of Life and Clinical/Economic Burden for Patients with Beta-Thalassemia



Summit, NJ – (Dec. 11, 2017) – Celgene Corporation today announced that data from two studies examining factors associated with patient experience in beta-thalassemia were presented at the 59th American Society of Hematology Annual Meeting between Dec. 9-12 in Atlanta, GA.

“Studies examining the experience of patients with beta-thalassemia presented at this year’s meeting show that challenges remain in both the transfusion-dependent and non-transfusion dependent settings,” said Maria Domenica Cappellini, M.D., of the University of Milan. “For transfusion-dependent patients, thalassemia represents a disease with a large economic burden. Also, while perception holds that non-transfusion dependent disease is less severe, the data presented here show that these patients suffer from worse quality of life compared with patients who are transfusion dependent.”

751 Quality of Life in Patients with β-Thalassemia: Transfusion Dependent Versus Non-Transfusion Dependent

In this study, patients with beta-thalassemia were prospectively enrolled in an observational study in Italy, Greece, Lebanon and Thailand and assessed for Quality of Life (QoL) by Short Form 36 Health Survey version 2 (SF-36v2) and Functional Assessment of Cancer Therapy (FACT)-Anemia (An) questionnaires both at baseline and then every three weeks. The analysis compared QoL between transfusion dependent (TD) (n=52) and non-transfusion dependent (NTD) (n=50) patients at enrollment in the study.

At study entry, all patients in the TD arm had received red-blood cell (RBC) transfusions within 24 weeks prior to enrolling, while 10% of NTD patients had RBC transfusions in the same period.

Patients with NTD beta-thalassemia had lower QoL on all assessments on the SF-36v2 questionnaire, with the exception of Role-Physical. Patients with NTD beta-thalassemia also experienced significantly lower QoL compared with TD patients in the areas of General Health (39.5 vs 44.0; P = 0.01), Vitality (49.3 vs 53.7; P = 0.01), and Mental Health (46.8 vs 50.8; P = 0.01), and in the Mental Component Summary Score (46.5 vs 50.8; P = 0.01). Similarly, patients with NTD beta-thalassemia reported worse QoL scores from the FACT-An questionnaire in all areas and significant differences were observed for Emotional Well-Being (18.5 vs 20.0; P = 0.02), Functional Well-Being (20.0 vs 23.2; P < 0.01), and FACT-General (82.9 vs 89.4; P = 0.01). 2095 Clinical and Economic Burden of Transfusion-Dependent β-Thalassemia in Adult Patients in the United States A second study focused on the health-economic impact of TD beta-thalassemia versus matched controls. In this retrospective analysis conducted from database information, utilization, outcomes and cost data from healthcare services in both inpatient and outpatient settings were reviewed. TD patients (n=50) had higher rates of diabetes (18% vs. 6%, P = 0.009) than controls (N=250). TD patients also had higher rates of cardiac disease (30% vs 2%, P < 0.001), hypogonadism (22% vs 7%, P = 0.002), osteoporosis (16% vs 1%, P < 0.001), and hypothyroidism (12% vs 4%, P = 0.045) compared with controls. TD patients required an average of 17 RBC transfusions per year, 23 days apart and had significantly higher outpatient visits compared to controls. Inpatients and ER visits were not significantly different between the groups. Average total healthcare costs in the TD group were $128,062 (± 62,260), compared to $5,438 (± 11.855) for controls (P < 0.001). Additionally, medical and medication costs were significantly higher for TD patients compared with controls (P < 0.001 in each case). Total costs for TD patients were primarily the result of iron chelation and transfusion costs. About Celgene Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation. For more information, please visit Follow Celgene on Social Media: @Celgene, Pinterest, LinkedIn, Facebook and YouTube.